I'm a Professor of Biomedical Engineering, Computer Science, and Biostatistics in the Institute of Genetic Medicine at Johns Hopkins University's School of Medicine. From 2005-2011 I was the Horvitz Professor of Computer Science and Director of the Center for Bioinformatics and Computational Biology at the University of Maryland, College Park. Before joining UMD, I was at The Institute for Genomic Research, where I sequenced the genomes of many bacteria, including those used in the 2001 anthrax attacks. At TIGR I was part of the Human Genome Project and the co-founder of the influenza virus sequencing project (which is when I first learned of the anti-vaccine movement). My research group develops software for DNA sequence analysis, and our (free) software is used by scientific laboratories around the globe. I did my B.A. and M.S. at Yale University, and my Ph.D. at Harvard University, and I have published over 200 scientific papers. Follow me on Facebook or Twitter (@stevensalzberg1), or just subscribe to my alternate blog, http://genome.fieldofscience.com.

A Breakthrough Cure for Leukemia?

How blood cells are created from stem cells. Image credit: National Cancer Institute.

This week a group of scientists from Memorial Sloan-Kettering Cancer Center published what may be a genuine breakthrough in the search for a cure for cancer. The simple word “cancer” disguises what is really hundreds of diseases, most caused by genetic mutations that make a cell start replicating out of control. The new study looks at a deadly form of leukemia, called B cell acute lymphoblastic leukemia, or B-ALL, and describes a radically new type of cancer therapy that uses genetic modification of a patient’s own cells.

First a bit of background. Leukemias are cancers of the blood, which means they don’t form solid tumors at all. Instead, the cancer cells circulate in the blood, going virtually everywhere in the body. The blood has many cell types within it, including white blood cells or leukocytes, which is the origin of the word leukemia. Lymphocytes are a type of white blood cell, and the two most common subtypes are called B cells and T cells. B-ALL, then, is a cancer where a B lymphocyte has suddenly turned cancerous.

Scientists have developed chemotherapies to treat B-ALL, but if the first line of treatments fail and the cancer returns, the patient faces a very grim prognosis. We really don’t have any effective treatments at this point, and most patients die.

The new leukemia therapy is a technological tour de force. The scientific team at Memorial Sloan-Kettering, led by Renier Brentjens and Michel Sadelain, genetically modified T cells from each patient so that these T-cells would target cancerous B cells. The modified T cells use something called a “chimeric antigen receptor,” or CAR, which they designed so that it would attach itself to a specific protein, called CD19, that sticks out of the surface of most cancerous B cells. (This technique was invented by another group at Memorial Sloan-Kettering, led by Isabelle Rivière.)

What was amazing about this study is that in all five patients, their cancers virtually disappeared in just a few weeks. As described in a The New York Times report, one of the sickest patients, 58-year-old David Aponte, saw his leukemia disappear in just eight days. But we cannot know for certain yet if the therapy alone is a cure. This is because the patients also had stem cell transplants within a few months after therapy, which may have helped eliminate any remaining cancer cells. The therapy itself was temporarily very toxic, requiring close monitoring and steroid treatments to lessen the toxic side effects. This may have been the reason that one patient died: the steroids may have hampered the therapy by destroying the genetically modified cells.

If this therapy were used as a first-line treatment of B-ALL, rather than in patients who have tried conventional therapy and relapsed, it might be even more effective (as the authors themselves suggest). In particular, it is likely to be less toxic in patients who are at earlier stages of the disease and have fewer tumor cells in their blood. Thus despite its promise, CAR T-cell therapy needs to be refined and tested further before we can declare it a success.

I’d like to make a final observation: this work, like almost all biomedical research in the U.S., was supported by the NIH, whose budget was just cut severely by Congress with its ill-conceived “sequester.” If we keep cutting biomedical research, we won’t see many more breakthroughs like this one. Even this dramatic result, promising though it is, needs more research to improve it and to test it on other leukemias, including chronic lymphycytic leukemia and non-Hodkins lymphoma, where it has already shown very promising results.

This leukemia treatment didn’t just appear out of thin air. It uses a technology that was invented four years ago, and that technology in turn is based on other discoveries that now make it possible to re-engineer a patient’s own cells and turn them into a treatment. Who knows where the next ten years will take us? Now is the time to be increasing our investment in biomedical research.

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Thank you Mr. Salzberg for posting. I just forwarded this info to a hematologist in France en route to a conference in Paris. This new therapy could be less toxic than R-CHOP and BEAM! As a Democrat Abroad member, I will definitely be submitting a letter to my Senator(NC) and Congress concerning biomedical research budget cuts.

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Leukemia and any other cancers on Earth can be erased from the face of the planet, once everybody (kids and adults) start doing my discovery – the PCK – The Personal Cancer Killer – the complete prevention and cure (for those now sick) for kids and adults of any diseases – from the common cold to cancer – just an exercise for a minute a day for prevention and for 2 – 3 minutes a day for the cure – for max. 30 days – no metastases or recurrences of any cancers are at all possible. Cancer Killer is by far more powerful than the immune system itself, keeps it intact all the time. No killer viruses, bacteria, germs and cancers on Earth got any chance against the tremendous power of the Cancer Killer – they just die the moment they touch you.

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please help my mother.. she was diagnosed with leukemia, and we don’t want to lose her.. we don’t have money to support her treatment, and I am willing to do whatever is needed to support her.. please, if anyone should see this, and you have some one who can help us, please help us.. I can’t tell you everything at the moment, it really shocked our family to know her status, but I’m begging for help.. you can contact me through my email — skullbocks.deck@gmail.com, and I’m from the Philippines.. you may also visit our house, and you’ll see that we don’t have much to support my mother.. please, help us, please.. I’m begging you..

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To Mr Salzberg. as i read you article, can you make my brother as one of you living evidence, he was diagnose as AML in year 2006, until last year 2013 he still being a healthy man, but after his first attack of the pneumonia his immune system become weaker day by day, he refuse to continue the chemotherapy process, now he tried to struggle on his own, but his condition become worst, i’m his donor for the bone marow traNSPLANT, I HOPE you could take my brother as one of your patient under treatment, you could test whether your biomedical research 100% works n can cure leukemia patient, do reply me if you could help us, my brother his only 38years old, i ‘m still hoping he will be cured always pray for him…,